On July 5, 2023, the Phase I/II clinical trial research center launch meeting for the intrathecal injection of GC101 injection, an AAV gene therapy drug independently developed by Beijing GeneCradle Technology Co., Ltd. (hereinafter referred to as "GeneCradle"), for the treatment of patients with type 2 spinal muscular atrophy (SMA) was successfully held at the lead unit, the Department of Pediatrics, Peking University First Hospital, with Professor Xiong Hui as the principal investigator.

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular genetic disease. The carrier frequency of its pathogenic gene in the Chinese population is about 1/42, and the incidence rate in newborns is about 1/10,000. It has been included in the "First List of Rare Diseases" jointly issued by the National Health Commission and other five departments. According to the severity of the disease and the age of onset, SMA is mainly divided into five phenotypes. Among them, most patients with type 2 SMA will lose the ability to sit alone and become paralyzed during adolescence, and patients often die from complications such as respiratory failure. There are currently three therapeutic drugs for SMA, nusinersen, lisapram and Zolgensma. The first two drugs have been approved for marketing in China and included in the medical insurance reimbursement list.

The advent of gene therapy provides a new way for long-term treatment of rare genetic diseases such as SMA. GC101 adeno-associated virus injection is a recombinant adeno-associated virus type 9 vector (rAAV9) carrying a normal SMN1 gene expression unit. It is administered once by intrathecal injection to express the SMN1 gene in motor neuron cells, thereby improving the function of affected cells such as motor neurons, and supplemented with respiratory and motor rehabilitation training to improve the respiratory and motor abilities of SMA patients.

At the meeting, the main researcher, Professor Xiong Hui, introduced the clinical team members participating in this clinical trial, and said that the pediatric neuromuscular disease team of Peking University First Hospital has rich experience in the diagnosis and treatment of rare diseases, especially SMA. Previously, SMA treatment has always been a foreign brand of drugs. This clinical trial will lead the team to uphold a scientific attitude and complete the trial project with high quality in accordance with GCP requirements, making it a clinical trial of domestic gene drugs that attracts worldwide attention, creating our domestic gene drugs, and benefiting the vast SMA patient population. This launch meeting also marks the first time that the scope of clinical trials of domestically developed SMA gene therapy drugs has been expanded to type 2 SMA patients. In the future, we will work with various units to promote type 2 SMA clinical trials and further improve the accessibility of gene therapy drugs for type 2 SMA patients.

The launch meeting was attended by clinical trial personnel from Peking University First Hospital, Dr. Dong Xiaoyan, co-founder of GeneCradle, Professor Zhu Zhiming, Vice President of Clinical Operations, Dr. Wang Xiaodong, Vice President of Medical Affairs, members of the drug clinical trial operations and medical team, representatives of contract research organizations, and representatives of the data statistics party. The participants introduced and discussed in detail the key contents of the research plan, clinical operation process, drug management and safety management, and fully implemented the details of the plan to ensure the smooth implementation of the trial plan.

I wish this clinical trial a complete success under the leadership of Director Xiong Hui, with the joint efforts of the clinical research team and research doctors from many well-known hospitals across the country, to bring long-term and better clinical treatment methods to patients with type 2 SMA, and transform gene therapy technology from scientific research to treatment and cure.

About GeneCradle

Beijing GeneCradle Technology Co., Ltd. is a national high-tech enterprise with AAV vector delivery technology-mediated gene therapy drug development as its core business. Its mission is to promote China's rare disease gene drugs from basic to clinical and market, benefiting patients and families. The company focuses on the development of gene therapy drugs in the fields of hereditary neuromuscular diseases, genetic metabolic diseases, lysosomal diseases and ophthalmic diseases. By promoting the development and clinical application of rare disease gene drugs, it has a deeper understanding of life and health, and has transitioned gene therapy technology and products from rare diseases to the treatment and rehabilitation of chronic diseases and other major diseases.